One key issue is the need to demonstrate that an orphan medicine has a significant benefit over existing treatments
Pharmaceutical firms have called on the European Commission to ensure that a planned review of the European Union (EU) orphan drug regulation allows enough flexibility in the legal definition of such medicines to enable manufacturers to effectively use its fast-track market authorisation.
Key comments have come in a public consultation on the proposals from a joint task force on orphan medicinal products and rare diseases between the European Federation of Pharmaceutical Industries and Associations (EFPIA) and the European Association for BioIndustries (EuropaBio).
Noting that the Commission had promised to avoid 'overly prescriptive requirements' preventing pharmaceutical firms from accessing the system, the EFPIA and EuropaBio argued that in drafts, Brussels had moved away from this understanding and 'seems more concerned by completeness rather than appropriateness of data requirements'.
The associations said: 'In the case of rare diseases, development can be complicated by a lack of scientific knowledge on the disease and by small patient populations. It is important to consider the feasibility of some of the data requirements.'
One key issue is the need to demonstrate that an orphan medicine has a significant benefit over existing treatments, and the two industry associations said pharmaceutical firms should be allowed to demonstrate this not just through direct comparative head-to-head studies. Historical comparison, indirect comparison, non-clinical endpoints and other methods should also be permitted, said the associations, as well as new adaptive trials designs or other innovations, such as trials-of-one.
The EFPIA and EuropaBio were also concerned that the revised regulation could put orphan drug status at risk when a manufacturer requested authorisation for an additional use and significant benefit could not be demonstrated. This would 'negatively impact companies’ investment decisions in rare disease research', warned the associations.
Other contribution were received from the UK BioIndustry Association (BIA); the Bundesverband der Pharmazeutischen Industrie (BPI – German Pharmaceutical Industry Association); and the European Generic Medicines Association (EGA).
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